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Displaying 1 - 8 of 8

Kids MoD PAH Trial

Condition: Lung Disease / Pulmonary Hypertension
Investigator: Usha Krishnan, MD
Status: Currently Recruiting
This study is being done to determine whether early combination therapy with two study drugs called sildenafil (REVATIO) and bosentan (TRACLEER) can improve pulmonary hypertension when compared to using sildenafil alone. Pediatric pulmonary arterial hypertension (PAH) is a severe medical problem that often worsens with time. It is caused by problems with…
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A study for infants with pre-symptomatic Spinal Muscular Atrophy (SMA)

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
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A study for patients with spinal muscular atrophy using study drug, ISIS 396443 to test safety and effectiveness

Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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A study for patients with pulmonary hypertension using study drug Riociguat

Condition: Lung Disease / Pulmonary Hypertension
Investigator: David Lederer, MD
Status: Closed
The purpose of this study is to study the effects of a new study drug known as Riociguat in people diagnosed with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP). Patients will be in the study for approximately 26 weeks in which they will take the pill Riogciguat three times a day.
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Investigation of an ultrasound device for pulmonary artery denervation in patients with pulmonary hypertension.

Condition: Lung Disease / Pulmonary Hypertension
Investigator: Erika Berman-Rosenzweig, MD
Status: Closed
This is a clinical research study designed to determine the safety and effectiveness of the TIVUS system, an investigational device used in the treatment of PAH. The TIVUS System is intended to treat patients with pulmonary artery hypertension by injuring the sympathetic nerves surrounding the pulmonary vasculature (denervation) using ultrasonic energy. The…
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The Autism Biorepository

Condition: Developmental Disorders / Autism
Investigator: Jeremy Veenstra-VanderWeele, MD
Status: Closed
The Translational Medicine Program at the Center for Autism and the Developing Brain is recruiting individuals with autism from 1 to 40 years of age and their biological family members from 1 - 70 years of age to participate in our Autism Biorepository. Participants will be given a travel reimbursement of $25 for 2 family members, $30 for 3 family members,…
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Rituximab for Systemic Sclerosis Associated Pulmonary Arterial Hypertension (SSc-PAH)

Condition: Lung Disease / Pulmonary Hypertension
Investigator: Elana Bernstein, MD
Status: Closed
This study involves two infusions of either rituximab or placebo, two right heart catheterizations (6 months apart), and a variety of other clinical study procedures and exams.
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The aV1ation Study

Condition: Developmental Disorders / Autism
Investigator: Jeremy Veenstra-VanderWeele, MD
Status: Closed
The Translational Medicine Program at the Center for Autism and the Developing Brain is recruiting individuals with autism from 5 -17 years of age with an IQ over 70 to participate in our study called aV1ation. The purpose of this study is to test an investigational medicine that blocks a hormone receptor in the brain linked to the control of socialization…
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